CO2020011068A2 - Métodos y composiciones para la administración de proteínas terapéuticas - Google Patents
Métodos y composiciones para la administración de proteínas terapéuticasInfo
- Publication number
- CO2020011068A2 CO2020011068A2 CONC2020/0011068A CO2020011068A CO2020011068A2 CO 2020011068 A2 CO2020011068 A2 CO 2020011068A2 CO 2020011068 A CO2020011068 A CO 2020011068A CO 2020011068 A2 CO2020011068 A2 CO 2020011068A2
- Authority
- CO
- Colombia
- Prior art keywords
- compositions
- methods
- therapeutic proteins
- administration
- cns
- Prior art date
Links
- 102000004169 proteins and genes Human genes 0.000 title abstract 4
- 108090000623 proteins and genes Proteins 0.000 title abstract 4
- 230000001225 therapeutic effect Effects 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 210000003169 central nervous system Anatomy 0.000 abstract 3
- 230000008499 blood brain barrier function Effects 0.000 abstract 2
- 210000001218 blood-brain barrier Anatomy 0.000 abstract 2
- 102000014914 Carrier Proteins Human genes 0.000 abstract 1
- 108010001857 Cell Surface Receptors Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 108091008324 binding proteins Proteins 0.000 abstract 1
- 208000037765 diseases and disorders Diseases 0.000 abstract 1
- 102000006240 membrane receptors Human genes 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
- A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
- A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
- A61K47/6815—Enzymes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6849—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6875—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody being a hybrid immunoglobulin
- A61K47/6879—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody being a hybrid immunoglobulin the immunoglobulin having two or more different antigen-binding sites, e.g. bispecific or multispecific immunoglobulin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6881—Cluster-antibody conjugates, i.e. the modifying agent consists of a plurality of antibodies covalently linked to each other or of different antigen-binding fragments covalently linked to each other
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
- A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2839—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the integrin superfamily
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2896—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/15—Humanized animals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Immunology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Gastroenterology & Hepatology (AREA)
- Inorganic Chemistry (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Peptides Or Proteins (AREA)
Abstract
Se describen composiciones y métodos para administrar una proteína terapéutica al sistema nervioso central (CNS), con el objetivo de tratar enfermedades y trastornos que afectan al CNS, tal como el tratamiento de enfermedades de almacenamiento lisosómico. Se proporcionan proteínas terapéuticas administradas a través de una cantidad terapéuticamente eficaz de una composición de nucleótido que codifica la proteína terapéutica conjugada a una proteína de unión a receptores de la superficie celular que cruza la barrera hematoencefálica (BBB).
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862627721P | 2018-02-07 | 2018-02-07 | |
US201862777683P | 2018-12-10 | 2018-12-10 | |
PCT/US2019/017116 WO2019157224A1 (en) | 2018-02-07 | 2019-02-07 | Methods and compositions for therapeutic protein delivery |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2020011068A2 true CO2020011068A2 (es) | 2020-12-10 |
Family
ID=65576681
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2020/0011068A CO2020011068A2 (es) | 2018-02-07 | 2020-09-04 | Métodos y composiciones para la administración de proteínas terapéuticas |
Country Status (16)
Country | Link |
---|---|
US (1) | US20200399623A1 (es) |
EP (1) | EP3749373A1 (es) |
JP (2) | JP2021512899A (es) |
KR (1) | KR20200118151A (es) |
CN (1) | CN112040985A (es) |
AU (1) | AU2019218892A1 (es) |
BR (1) | BR112020016005A2 (es) |
CA (1) | CA3090519A1 (es) |
CL (1) | CL2020002043A1 (es) |
CO (1) | CO2020011068A2 (es) |
IL (1) | IL276464A (es) |
MA (1) | MA51796A (es) |
MX (1) | MX2020008274A (es) |
PH (1) | PH12020551180A1 (es) |
SG (1) | SG11202007363TA (es) |
WO (1) | WO2019157224A1 (es) |
Families Citing this family (23)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
HUE052155T2 (hu) * | 2015-12-08 | 2021-04-28 | Regeneron Pharma | Enzimek internalizálására szolgáló készítmények és módszerek |
AU2018281280A1 (en) | 2017-06-07 | 2020-01-16 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
AU2019209770A1 (en) | 2018-01-16 | 2020-09-03 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity |
WO2020005341A1 (en) * | 2018-06-29 | 2020-01-02 | Icahn School Of Medicine At Mount Sinai | Anc80 encoding sphingolipid-metabolizing proteins |
US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
CA3159018A1 (en) * | 2019-11-19 | 2021-05-27 | Michael W. O'CALLAGHAN | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
WO2022150609A1 (en) * | 2021-01-08 | 2022-07-14 | Cls Therapeutics Limited | Combination of deoxyribonuclease enzyme and cell therapies for treatment of cancer |
WO2022226313A1 (en) * | 2021-04-23 | 2022-10-27 | University Of Cincinnati | Therapeutics for hyponatremia and polycystic kidney disease |
US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
CA3233698A1 (en) * | 2021-11-04 | 2023-05-11 | Regeneron Pharmaceuticals, Inc. | Viral particles retargeted to skeletal muscle |
US20230220100A1 (en) | 2022-01-10 | 2023-07-13 | Regeneron Pharmaceuticals, Inc. | Bbb-targeted gaa delivered as gene therapy treats cns and muscle in pompe disease model mice |
TW202332767A (zh) | 2022-02-02 | 2023-08-16 | 美商雷傑納榮製藥公司 | 用於治療龐貝氏症之抗TfR:GAA及抗CD63:GAA插入 |
WO2023187728A1 (en) * | 2022-04-01 | 2023-10-05 | Takeda Pharmaceutical Company Limited | Gene therapy for diseases with cns manifestations |
US11931421B2 (en) | 2022-04-15 | 2024-03-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
WO2024026488A2 (en) | 2022-07-29 | 2024-02-01 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a modified transferrin receptor locus |
US20240139329A1 (en) * | 2022-10-27 | 2024-05-02 | California Institute Of Technology | Targets for Receptor-Mediated Control of Therapeutic Biodistribution and Efficacy |
Family Cites Families (64)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US543153A (en) | 1895-07-23 | kidder | ||
EP0740702A1 (en) | 1992-12-02 | 1996-11-06 | Zymogenetics, Inc. | Novel human amyloid protein precursor homologue and kunitz-type inhibitors |
US5436153A (en) | 1992-12-02 | 1995-07-25 | Sprecher; Cindy A. | Human amyloid protein precursor homolog and Kunitz-type inhibitor |
US5585108A (en) | 1994-12-30 | 1996-12-17 | Nanosystems L.L.C. | Formulations of oral gastrointestinal therapeutic agents in combination with pharmaceutically acceptable clays |
WO1997005266A1 (en) | 1995-07-25 | 1997-02-13 | Introgene B.V. | Methods and means for targeted gene delivery |
US6265389B1 (en) | 1995-08-31 | 2001-07-24 | Alkermes Controlled Therapeutics, Inc. | Microencapsulation and sustained release of oligonucleotides |
DK1068238T3 (da) | 1998-04-02 | 2013-01-14 | Xintela Ab | Integrinheterodimer og subunit deraf |
US6649138B2 (en) | 2000-10-13 | 2003-11-18 | Quantum Dot Corporation | Surface-modified semiconductive and metallic nanoparticles having enhanced dispersibility in aqueous media |
CA2467763C (en) | 2001-12-21 | 2011-09-13 | Alcon, Inc. | Use of synthetic inorganic nanoparticles as carriers for ophthalmic and otic drugs |
SE0301087D0 (sv) | 2003-04-14 | 2003-04-14 | Cartela Ab | New monoclonal antibody |
AU2004317548A1 (en) | 2004-03-25 | 2005-10-06 | Centocor, Inc. | Use of EMMPRIN antagonists for the treatment of diseases associated with excessive angiogenesis |
US20110182805A1 (en) | 2005-06-17 | 2011-07-28 | Desimone Joseph M | Nanoparticle fabrication methods, systems, and materials |
EP4332227A1 (en) | 2005-08-23 | 2024-03-06 | The Trustees of the University of Pennsylvania | Rna containing modified nucleosides and methods of use thereof |
GB0615662D0 (en) | 2006-08-07 | 2006-09-13 | Affitech As | Antibody |
JP5901877B2 (ja) | 2007-07-27 | 2016-04-13 | アーメイゲン・テクノロジーズ・インコーポレイテッドArmagen Technologies, Inc. | 中枢神経系のα−L−イデュロニダーゼ活性を増加させるための方法および組成物 |
JP5564049B2 (ja) | 2008-09-29 | 2014-07-30 | ヤンセン バイオテツク,インコーポレーテツド | 抗cd147抗体、方法及び使用 |
JP2012532455A (ja) | 2009-07-01 | 2012-12-13 | ケメット エレクトロニクス コーポレーション | 高電圧能力を有する高静電容量の多層 |
WO2011012316A2 (de) | 2009-07-31 | 2011-02-03 | Ludwig-Maximilians-Universität | Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression |
EP2332995A1 (en) | 2009-12-10 | 2011-06-15 | Bayer Schering Pharma Aktiengesellschaft | Neutralizing prolactin receptor antibodies and their therapeutic use |
US20110223176A1 (en) | 2010-03-11 | 2011-09-15 | Abbott Laboratories | Basigin binding proteins |
US9782342B2 (en) | 2010-10-11 | 2017-10-10 | Wichita State University | Composite magnetic nanoparticle drug delivery system |
TWI689314B (zh) | 2010-11-30 | 2020-04-01 | 建南德克公司 | 低親和力血腦障壁受體抗體及其用途 |
US20140212503A1 (en) | 2011-03-17 | 2014-07-31 | Hyukjin Lee | Delivery system |
WO2012136519A1 (en) | 2011-04-06 | 2012-10-11 | Bayer Pharma Aktiengesellschaft | Use of neutralizing prolactin receptor antibodies for the treatment and prevention of antiestrogen-resistant breast cancer |
US20120283503A1 (en) | 2011-04-29 | 2012-11-08 | The Johns Hopkins University | Nanoparticle loaded stem cells and their use in mri guided hyperthermia |
EP2530089A1 (en) | 2011-06-03 | 2012-12-05 | Bayer Pharma Aktiengesellschaft | Neutralising prolactin receptor antibody Mat3 and its therapeutical use |
RU2014124530A (ru) | 2011-11-23 | 2015-12-27 | Идженика, Инк. | Антитела к cd98 и способы их применения |
WO2013081706A1 (en) | 2011-12-02 | 2013-06-06 | Armagen Technologies, Inc. | Methods and compositions for increasing arylsulfatase a activity in the cns |
US8883979B2 (en) | 2012-08-31 | 2014-11-11 | Bayer Healthcare Llc | Anti-prolactin receptor antibody formulations |
JP2016515515A (ja) | 2013-03-15 | 2016-05-30 | バイエル・ヘルスケア・エルエルシーBayer HealthCare LLC | 抗プロラクチン受容体抗体製剤 |
US9738717B2 (en) | 2013-05-15 | 2017-08-22 | The Translational Genomics Research Institute | Hybridoma clones, monoclonal antibodies, and methods of use |
TWI573805B (zh) | 2013-05-20 | 2017-03-11 | 建南德克公司 | 抗轉鐵蛋白受體抗體及其使用方法 |
US10906981B2 (en) | 2013-07-19 | 2021-02-02 | The Regents Of The University Of California | Compositions and methods related to structures that cross the blood brain barrier |
US9545451B2 (en) | 2013-08-21 | 2017-01-17 | Regeneron Pharmaceuticals, Inc. | Anti-PRLR antibodies and methods for killing PRLR-expressing cells |
TWI641620B (zh) | 2013-08-21 | 2018-11-21 | 再生元醫藥公司 | 抗-prlr抗體及其用途 |
JP6476194B2 (ja) | 2014-01-03 | 2019-02-27 | エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft | 二重特異性抗ハプテン/抗血液脳関門受容体抗体、それらの複合体、及び血液脳関門シャトルとしてのそれらの使用 |
RU2694659C2 (ru) | 2014-01-06 | 2019-07-16 | Ф. Хоффманн-Ля Рош Аг | Одновалентные модули-переносчики через гематоэнцефалический барьер |
US9629801B2 (en) | 2014-01-10 | 2017-04-25 | Wisconsin Alumni Research Foundation | Blood-brain barrier targeting antibodies |
JP2017114763A (ja) | 2014-03-26 | 2017-06-29 | 第一三共株式会社 | 抗cd98抗体−薬物コンジュゲート |
CN106573074B (zh) | 2014-06-02 | 2022-04-12 | 里珍纳龙药品有限公司 | 生物活性分子偶联物、试剂和制备方法及其治疗用途 |
EP3218411B1 (en) * | 2014-11-14 | 2022-01-12 | Ossianix, Inc. | Variable new antigen receptors (vnars) directed against transferrin receptor (tfr) and their use |
EP3221362B1 (en) | 2014-11-19 | 2019-07-24 | F.Hoffmann-La Roche Ag | Anti-transferrin receptor antibodies and methods of use |
CN107250158B (zh) | 2014-11-19 | 2022-03-25 | 基因泰克公司 | 抗转铁蛋白受体/抗bace1多特异性抗体和使用方法 |
MA41022A (fr) * | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
WO2016094566A2 (en) | 2014-12-10 | 2016-06-16 | Genentech, Inc. | Blood brain barrier receptor antibodies and methods of use |
US10538589B2 (en) | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
LT3313879T (lt) | 2015-06-24 | 2022-03-25 | F. Hoffmann-La Roche Ag | Antikūnai prieš transferino receptorių su pritaikytu giminingumu |
PT3336185T (pt) | 2015-08-10 | 2022-01-27 | Univ Osaka | Anticorpo |
WO2017058944A1 (en) | 2015-09-29 | 2017-04-06 | Amgen Inc. | Asgr inhibitors |
AR106189A1 (es) | 2015-10-02 | 2017-12-20 | Hoffmann La Roche | ANTICUERPOS BIESPECÍFICOS CONTRA EL A-b HUMANO Y EL RECEPTOR DE TRANSFERRINA HUMANO Y MÉTODOS DE USO |
TWI819458B (zh) | 2015-10-02 | 2023-10-21 | 瑞士商赫孚孟拉羅股份公司 | 雙特異性抗‐人類cd20/人類轉鐵蛋白受體抗體及使用方法 |
HUE052155T2 (hu) * | 2015-12-08 | 2021-04-28 | Regeneron Pharma | Enzimek internalizálására szolgáló készítmények és módszerek |
US10233252B2 (en) | 2015-12-21 | 2019-03-19 | Wisconsin Alumni Research Foundation | pH-dependent antibodies targeting the transferrin receptor and methods of use thereof to deliver a therapeutic agent |
US20190352423A1 (en) * | 2016-02-05 | 2019-11-21 | Genmab A/S | Multispecific antigen-binding molecule with improved internalization characteristics |
CN105820250B (zh) | 2016-04-29 | 2019-04-30 | 中国人民解放军第四军医大学 | 一种抗basigin人源化抗体及其应用 |
BR112018075644A2 (pt) | 2016-06-08 | 2019-04-09 | Abbvie Inc. | anticorpos anti-cd98 e conjugados de anticorpo e fármaco |
CA3027047A1 (en) | 2016-06-08 | 2017-12-14 | Abbvie Inc. | Anti-cd98 antibodies and antibody drug conjugates |
BR112018075630A2 (pt) | 2016-06-08 | 2019-03-19 | Abbvie Inc. | anticorpos anti-cd98 e conjugados de fármaco de anticorpo |
CN110382534B (zh) | 2016-11-29 | 2023-05-09 | 里珍纳龙药品有限公司 | 治疗prlr阳性乳腺癌的方法 |
LT3573630T (lt) | 2017-01-27 | 2021-05-25 | Xintela Ab | Kaulų ir kremzlių pažeidimų arba ligų prevencija ir gydymas |
WO2018165619A1 (en) | 2017-03-09 | 2018-09-13 | Cytomx Therapeutics, Inc. | Cd147 antibodies, activatable cd147 antibodies, and methods of making and use thereof |
WO2018210898A1 (en) | 2017-05-18 | 2018-11-22 | F. Hoffmann-La Roche Ag | Reduction of application-related side reaction of a therapeutic antibody |
AU2018281280A1 (en) * | 2017-06-07 | 2020-01-16 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
CA3069193A1 (en) | 2017-07-10 | 2019-01-17 | Bayer Pharma Aktiengesellschaft | Prolactin receptor antibody for male and female pattern hair loss |
-
2019
- 2019-02-07 BR BR112020016005-9A patent/BR112020016005A2/pt unknown
- 2019-02-07 KR KR1020207025644A patent/KR20200118151A/ko unknown
- 2019-02-07 CN CN201980022383.3A patent/CN112040985A/zh active Pending
- 2019-02-07 US US16/968,452 patent/US20200399623A1/en active Pending
- 2019-02-07 EP EP19707943.7A patent/EP3749373A1/en active Pending
- 2019-02-07 CA CA3090519A patent/CA3090519A1/en active Pending
- 2019-02-07 AU AU2019218892A patent/AU2019218892A1/en active Pending
- 2019-02-07 MA MA051796A patent/MA51796A/fr unknown
- 2019-02-07 SG SG11202007363TA patent/SG11202007363TA/en unknown
- 2019-02-07 WO PCT/US2019/017116 patent/WO2019157224A1/en unknown
- 2019-02-07 MX MX2020008274A patent/MX2020008274A/es unknown
- 2019-02-07 JP JP2020542598A patent/JP2021512899A/ja active Pending
-
2020
- 2020-08-03 IL IL276464A patent/IL276464A/en unknown
- 2020-08-04 PH PH12020551180A patent/PH12020551180A1/en unknown
- 2020-08-06 CL CL2020002043A patent/CL2020002043A1/es unknown
- 2020-09-04 CO CONC2020/0011068A patent/CO2020011068A2/es unknown
-
2023
- 2023-02-22 JP JP2023026217A patent/JP2023054256A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
BR112020016005A2 (pt) | 2020-12-15 |
JP2021512899A (ja) | 2021-05-20 |
MA51796A (fr) | 2020-12-16 |
PH12020551180A1 (en) | 2021-06-07 |
WO2019157224A1 (en) | 2019-08-15 |
SG11202007363TA (en) | 2020-08-28 |
CN112040985A (zh) | 2020-12-04 |
CL2020002043A1 (es) | 2021-07-23 |
MX2020008274A (es) | 2020-11-11 |
IL276464A (en) | 2020-09-30 |
CA3090519A1 (en) | 2019-08-15 |
RU2020129184A (ru) | 2022-03-09 |
EP3749373A1 (en) | 2020-12-16 |
JP2023054256A (ja) | 2023-04-13 |
US20200399623A1 (en) | 2020-12-24 |
KR20200118151A (ko) | 2020-10-14 |
AU2019218892A1 (en) | 2020-10-01 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CO2020011068A2 (es) | Métodos y composiciones para la administración de proteínas terapéuticas | |
MX2009002893A (es) | Tratamiento de trastornos hepaticos mediante la administracion de conjugados de la proteina asociada al receptor (rap). | |
MX2009011473A (es) | Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. | |
MA41932B1 (fr) | Méthodes de traitement ou de prévention de migraines | |
CL2008000647A1 (es) | Compuestos derivados de indazol; composicion farmaceutica; yuso en el tratamiento de enfermedades tales como dolor, trastornos inflamatorios cronicos, trastornos del snc, entre otros. | |
UA92497C2 (ru) | Составы, которые содержат пептиды, стимулирующие рецептор эритропоэтина, и их применение | |
WO2013158680A3 (en) | Compositions and methods comprising celecoxib or related compounds and dextromethorphan | |
MX2019013690A (es) | Conjugados de farmaco-proteina con ciclodextrina. | |
EP4276180A3 (en) | Clostridium histolyticum enzyme | |
WO2012092458A3 (en) | Compositions comprising placental stem cells and platelet rich plasma, and methods of use thereof | |
GT200500332A (es) | Azabenzoxazoles para el tratamiento de trastornos del snc | |
WO2005000215A3 (en) | Methods for treating pain | |
PH12016502447A1 (en) | Cholix toxin-derived fusion molecules for oral delivery of biologically active cargo | |
WO2010003268A3 (en) | Methods and compositions for enhanced delivery of macromolecules | |
MX2018006299A (es) | Analogos de peptido de la hormona de estimulacion de alfa-melanocito. | |
MX2021005346A (es) | Constructos de suministro para transcitosis y metodos relacionados. | |
MX2019000547A (es) | Formulaciones de adamts13 liquidas y liofilizadas, estabilizadas. | |
TN2012000393A1 (en) | Agonist dr5 binding polypeptides | |
MX2021008114A (es) | Enlazadores sin rastro y conjugados de proteinas de los mismos. | |
WO2012177653A3 (en) | Targeting the neuromuscular junction for treatment | |
WO2005046593A3 (en) | Methods and compositions using thalidomide for the treatment and management of cancers and other diseases. | |
MX2023008330A (es) | Agonistas del receptor dual gip/glp-1 de molécula pequeña, composiciones farmacéuticas y preparación de las mismas para usarse en el tratamiento de trastornos metabólicos y hepáticos. | |
BR112012022209A2 (pt) | composições farmacêuticas compreendendo monoterpenos | |
GB2537783A (en) | Methods and compositions used in treating inflammatory and autoimmune diseases | |
WO2016205397A3 (en) | Target-specific delivery of therapeutic agents |